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FDA Morale in the New Era of Deregulation

5/4/2017 / by Dr. Evan Siegel  


Effects on FDA Morale and the Potential for en masse Resignations of that Agency’s Reviewers

In a previous blog we identified several critical processes that could be affected if there were major changes in the laws underpinning FDA review and approval of new drugs, biological therapies and vaccines.

Should Congress make major modifications in the Food, Drug and Cosmetics and Public Health Service Acts, the requirement for substantial evidence of both efficacy and safety could be altered. The resulting shift in assessment of risk/benefit could have a profound effect on the organization, procedures, internal guidance, workload, and approach of Agency Reviewers.

Over the last several years these Reviewers have increasingly been hired by the FDA from industry, university research, and the practice of medicine, thus incorporating their experience outside of the government environment and enriching the Agency with sophisticated, experienced, realistic and sound resources. Along with the traditional facilitated approaches to accelerating the medical product review process (e.g., Fast Track, Accelerated Approval, and Orphan Drug Designation), newer programs such as Breakthrough Designation and Regenerative Medicine Advanced Therapy Designation have responded to critical healthcare emergencies and added to the officially sanctioned means for bringing new therapeutic interventions to patients with unmet medical need, while still meeting well-defined standards for safety and efficacy.

We believe that a radical change in the requirements for substantial evidence of both safety and efficacy, if introduced into the FDA, will lead to a massive upheaval in both the morale of Agency Reviewers, and concerns for the overall health of the system for development, review and approval of new diagnostic and therapeutic interventions.

This could also have a significant adverse effect both on the US system for reimbursement and worldwide due to the fallout over the lowered competitiveness of products approved or licensed under inferior standards of risk/benefit. The FDA has often been thought of as a “gold standard” for review of new chemical and biological entities, emulated by regulatory authorities worldwide. Should the US system of substantial evidence of both efficacy and safety imposed prior to marketing approval of new products be compromised, ex-US regulators and reimbursement authorities could well place such products on a second or third tier for accessibility, thus adversely affecting US firms who have developed them under the new legal/regulatory scheme.

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Why Changes MUST Go Through Congress

4/6/2017 / by Dr. Evan Siegel  


The Food, Drug and Cosmetic Act and Public Health Services Act; Must be Changed through new Law in Congress

Last week, we identified a number of areas that would be impacted if there were major changes in how the FDA approved marketing applications.

The first area of impact is the 1962 amendments to the Food, Drug, and Cosmetic Act and the Public Health Service Act. Laws and regulations would need to be changed to result in functional differences in how drugs are evaluated by FDA (an Executive Order can’t do it!)

Congressional approval of one or more laws that would modify or void these Acts is necessary prior to any move to change the FDA review and approval process as well as industry strategies, expenditures and timeframes for moving new products to market.

Some changes could radically alter the risk/benefit profile of drugs and biologicals in the US, with fallout around the world. Nonclinical pharmacology support for clinical trials and the efficacy endpoints for clinical trials themselves could be massively reduced in type and scope, thus leading to uncertainty for patients being treated as well as the healthcare professionals who prescribe the affected products. Although modifications of these requirements could potentially allow for earlier availability of therapeutics to patients, they could also dramatically shift patient exposure to ineffective products, thus sharply reducing their usefulness and cost-benefit and disrupting the financing, resourcing, and eventual success for companies and other Sponsors who discover and develop these products.

The FDA has many programs for accelerated development and patient access to therapeutics that are showing both safety and efficacy potential. These include the treatment IND, single patient compassionate use, emergency IND, EUA, accelerated approval, breakthrough therapy and other designations. These programs have worked well in accelerating the product development process and allowing earlier access to experimental drugs and biologics. A fine-tuning of the system, with evolutionary changes in law, regulation and data requirements, that incrementally impact our entire system of therapeutics is a logical approach to providing access to SAFE AND EFFECTIVE therapies.